… diagnosed with a rare disease, he started ProQR to develop RNA therapies for rare diseases. Before founding ProQR, … for the ADAR enzymes responsible for adenosine to inosine RNAediting in humans. In addition, his group has led in the …
ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need.
